May 19 - 21, 2026

Boston Convention & Exhibition Center | Boston, MA

The World’s Most Important Orphan Drug & Rare Disease Event

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280+

Speakers

2,000+

Attendees

100+

Exhibitors

16

Streams of Content

Breaking barriers & fostering collaboration to help the rare disease community

The World Orphan Drug Congress USA brings together the leading pharmaceutical and biotechnology companies, government and regulators, patient advocates, payers, investors, and solution providers – all who all depend on our event to convene and brainstorm ways to advance

Orphan Drug development and improve access to life-saving therapies for rare disease patients.

Join the world’s defining event for the Orphan Drug sector and rare disease community May 19-21, 2026.

SECURE YOUR SPOT – REGISTER NOW

This is your chance to explore the industry’s latest innovations and tech + catch the most up-to-date, keynote-driven content.

Your ticket provides access to the premium conference, exhibition and networking opportunities with thousands of industry leaders

THE CONFERENCE

Hear about the latest developments in the rare disease and orphan drug space from top thought leaders in the industry.

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THE EXHIBITION

Discover innovative products and solutions revolutionizing the future of rare disease.

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NETWORKING

Connect with fellow rare disease and orphan drug advocates from around the world – all with a shared passion to improve the lives of rare disease patients.

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Sponsor the World Orphan Drug Congress USA

Get your product or solution in front of the leaders in the rare disease industry — from pharma, biotechs, governments, payers, investors and patient/patient advocates.

The World Orphan Drug Congress USA is a place where innovation and expertise is showcased, solutions are found and learning is done.

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pharma, biotechs, governments, payers, investors and patient patient advocates.

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Highlighted Speakers

Peter Marks
Director, Center For Biologics Evaluation And Research (CBER)
U.S. Food and Drug Administration

Peter Marks is speaking at World Orphan Drug Congress USA

Amy Nicole Nayar
VP of U.S. Patient Advocacy & Government Affairs
Novartis Gene Therapies

Amy Nicole Nayar is speaking at World Orphan Drug Congress USA

Diego Guarin
Executive Director, Regional Market Access
Merck

Diego Guarin is speaking at World Orphan Drug Congress USA

PJ Brooks
Deputy Director, NCATS Division of Rare Diseases Research Innovation
National Institutes of Health

PJ Brooks is speaking at World Orphan Drug Congress USA

Durhane Wong-Rieger
President, CEO
Canadian Organisation for Rare Disorder & Rare Diseases International

Durhane Wong-Rieger speaking at World Orphan Drug Congress USA

Melanie Lendnal
SVP, Policy & Advocacy
ALS Association

Melanie Lendnal speaking at World Orphan Drug Congress USA

Tim Hunt
CEO
Alliance for Regenerative Medicine

Tim Hunt speaking at World Orphan Drug Congress USA

Francis Pang
SVP, Global Market Access & Geographic Expansion
Orchard Therapeutics

Francis Pang speaking at World Orphan Drug Congress USA

Dionne Stalling
Executive Director
Rare & Black

Dionne Stalling speaking at World Orphan Drug Congress USA

Kinnari Patel
President, Head of R&D and COO
Rocket Pharma

Kinnari Patel speaking at World Orphan Drug Congress USA

VIEW OUR PAST SPEAKERS

Highlighted Speakers

Peter Marks
Director, Center For Biologics Evaluation And Research (CBER)
U.S. Food and Drug Administration

Sarah Glass
Chief Operating Officer
n-Lorem Foundation

Sarah Glass is speaking at World Orphan Drug Congress USA

Paul Aliu
Head Global Governance Office, Novartis Chief Medical Office (CMO)
Novartis

Paul Aliu is speaking at World Orphan Drug Congress USA

PJ Brooks
Acting Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS)
National Institutes of Health

Annie Kennedy
Chief of Policy, Advocacy, & Patient Engagement
EveryLife Foundation for Rare Diseases

Annie Kennedy speaking at World Orphan Drug Congress USA

Alexander Natz
Director General
EUCOPE - European Confederation of Pharmaceutical Entrepreneurs

Alexander Natz speaking at World Orphan Drug Congress USA

Durhane Wong-Rieger
President, Chief Executive Officer & Chair
Canadian Organisation for Rare Diseases & Rare Diseases International

Mary Wang
Programme Director
Rare Diseases International

Mary Wang speaking at World Orphan Drug Congress USA

Emil Kakkis
Chief Executive Officer
Ultragenyx Pharmaceutical

Emil Kakkis speaking at World Orphan Drug Congress USA

Peter Saltonstall
Chief Executive Officer
National Organization for Rare Disorders (NORD)

Peter Saltonstall speaking at World Orphan Drug Congress USA

VIEW OUR PAST SPEAKERS

The Start-to-Finish of Orphan Drugs
From regulation and policy to global pricing and gene therapy

PATIENT DATA

With so few patients, patient data/real-world-data (RWD/RWE) is the key element to advancing the rare disease space. What are best practices right now for data collection, ownership and sharing to benefit rare disease diagnosis, drug development and access.

NEXT GENERATION THERAPIES

The rare disease space is a breeding ground for innovative approaches in drug development and platform technologies. What are the products and approaches that will be making headlines in 5-10 years from now?

payers PAYERS

PAYERS

US-based insurers and single payer agencies around the world have a lot of impact of access and reimbursement for orphan products. Hear from top US and international payers on their strategies for orphan drugs.

CLINICAL DEVELOPMENT & REGULATORY

Understanding the regulatory and clinical landscape for rare disease drug development. How can companies, regulators and patient advocates work together to further patient voice, real world evidence, and decentralized clinical trials.

COMMERCIAL

What are the best commercial strategies for orphan products? How can companies bring products to new markets, build more efficient commercial operations, and develop effective launch strategies?

RARE DISEASE ADVOCACY WORLD

It all starts and ends with patients. So what are the challenges facing rare disease patients and patient organizations today? How can rare disease patient groups rethink funding strategies, partnerships with industry and advance policies around access?

global marketing & patient access GLOBAL MARKET & PATIENT ACCESS

GLOBAL MARKET & PATIENT ACCESS

Access is often the crucial and challenges last step for rare disease patients and developers. How can all stakeholders work together to ensure rare disease patients have access to appropriate treatments globally.

PRICING & REIMBURSEMENT

Pricing orphan products, especially one-time treatments, is complex and a key element to ensuring timely access for rare disease patients. What are the current best practices for pricing orphan products, negotiating innovative contracts and working with payers around the world?

PITCH & PARTNER

The orphan drug space is booming with innovation, M&A activity and partnerships. We play match maker for up and coming biotechs in the rare space and investors & pharma partners looking for their next blockbuster.

advanced therapies manufacturing ADVANCED THERAPIES - MANUFACTURING

ADVANCED THERAPIES - MANUFACTURING

What are the current roadblocks in manufacturing cell and gene therapies and how can companies efficiently scale-up for commercial production?

advanced therapies clinical development ADVANCED THERAPIES - CLINICAL DEVELOPMENT

ADVANCED THERAPIES - CLINICAL DEVELOPMENT

The regulatory and clinical development landscape is ever evolving for cell and gene therapies. How are companies ensuring continued development of these potentially transformative products and what clinical hurdles remain?

RARE ONCOLOGY

As the rare cancer field evolves and diagnosis becomes more precise, how can all stakeholders work together to advance drug development for rare cancer patients?

advanced therapies commercialization ADVANCED THERAPIES - COMMERCIALIZATION

ADVANCED THERAPIES - COMMERCIALIZATION

Rare disease patients are uniquely positioned to benefit from the advancements of cell and gene therapies. What are the best strategies for commercializing these innovative products and how do health systems need to evolve to accommodate patient access and reimbursement?

DIGITAL HEALTH & ARTIFICIAL INTELLIGENCE

Approaches in AI and digital health span the full development life cycle of drug development and commercialization. What are the best approaches to implementing these technologies in patient finding, clinical development and commercialization for rare diseases?

diagonis DIAGNOSIS

DIAGNOSIS

It takes an average of seven years for an accurate diagnosis. How can all stakeholders broaden access to Newborn Screening, Whole Genome Sequencing and Genetic testing to shorten the diagnostic odyssey for rare disease patients?

DISCOVERY

Technologies and processes are ever. How is the growing access to data, and use of AI and machine learning impacting drug discovery in the rare disease space?

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COMPLIMENTARY VIP ACCESS for
Patient Groups, Healthcare workers & Investors!

Apply for your free pass below to secure your spot

apply now

Networking Is Our Sweet Spot

By App. By Facilitated Meetings. By RoundTables. By Hangin’ Out.

So you can: Exchange ideas, Build brand, Form friendships and partnerships, Grow your professional network, Explore the future, Uncover new opportunities.

FIND OUT MORE

"If there is one event you want to go to as a stakeholder in rare diseases, it is the World Orphan Drug Congress USA. This conference has helped to define the world of rare diseases, it’s not just a leading event, it has actually helped create the future for rare diseases"

President And Chief Executive Officer, Canadian Organization For Rare Disorders

"The support, kindness, courtesy and interest shown by WODC Team in our company’s drug development program for orphan disease glioblastoma, to aid us to secure funding to run registration trials"

Chief Executive Officer, Tactical Therapeutics Inc

The World’s Most Important Orphan Drug & Rare Disease Event

280+

2,000+

100+

16

Breaking barriers & fostering collaboration to help the rare disease community

This is your chance to explore the industry’s latest innovations and tech + catch the most up-to-date, keynote-driven content.

Your ticket provides access to the premium conference, exhibition and networking opportunities with thousands of industry leaders

Sponsor the World Orphan Drug Congress USA

2025 Sponsors & Exhibitors

Highlighted Speakers

Highlighted Speakers

The Start-to-Finish of Orphan Drugs
From regulation and policy to global pricing and gene therapy

COMPLIMENTARY VIP ACCESS for
Patient Groups, Healthcare workers & Investors!

Apply for your free pass below to secure your spot

Networking Is Our Sweet Spot

Join us in Boston

May 19-21, 2026

Get Involved At World Orphan Drug Congress USA

Connect with us

To Sponsor Or Exhibit

Justin Franks
justin.franks@terrapinn.com
t/ +1 914 819 3506

To Speak

Kaylee Nguyen
kaylee.nguyen@terrapinn.com

Marketing & Press

Kaylie Brogan
kaylie.brogan@terrapinn.com

Created by

The World’s Most Important Orphan Drug & Rare Disease Event

280+

2,000+

100+

16

Breaking barriers & fostering collaboration to help the rare disease community

This is your chance to explore the industry’s latest innovations and tech + catch the most up-to-date, keynote-driven content.

Your ticket provides access to the premium conference, exhibition and networking opportunities with thousands of industry leaders

Sponsor the World Orphan Drug Congress USA

2025 Sponsors & Exhibitors

Highlighted Speakers

Highlighted Speakers

The Start-to-Finish of Orphan Drugs From regulation and policy to global pricing and gene therapy

COMPLIMENTARY VIP ACCESS forPatient Groups, Healthcare workers & Investors!

Apply for your free pass below to secure your spot

Networking Is Our Sweet Spot

Join us in Boston

May 19-21, 2026

Get Involved At World Orphan Drug Congress USA

Connect with us

To Sponsor Or Exhibit

Justin Franks justin.franks@terrapinn.com t/ +1 914 819 3506

To Speak

Kaylee Nguyen kaylee.nguyen@terrapinn.com

Marketing & Press

Kaylie Brogan kaylie.brogan@terrapinn.com

Created by

The Start-to-Finish of Orphan Drugs
From regulation and policy to global pricing and gene therapy

COMPLIMENTARY VIP ACCESS for
Patient Groups, Healthcare workers & Investors!

Justin Franks
justin.franks@terrapinn.com
t/ +1 914 819 3506

Kaylee Nguyen
kaylee.nguyen@terrapinn.com

Kaylie Brogan
kaylie.brogan@terrapinn.com